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AAP Makes Recommendations on Use of Off-Label Drugs for Children

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​Less than half of medications include specific labeling for children, which means pediatricians often must decide what is appropriate to prescribe based on their clinical judgment. In a revised policy statement, the American Academy of Pediatrics (AAP) offers guidance to physicians using drugs off-label.

The policy statement, “Off-Label Use of Drugs in Children,” published in the March 2014 Pediatrics (released online Feb. 24), updates a statement that was published in 2002 and reaffirmed in 2005.

“Pediatricians must prescribe drugs off-label, simply because an overwhelming number of critical drugs still have no information on the label for use in children,” said Kathleen Neville, MD, FAAP, lead author of the policy statement and chair of the AAP Committee on Drugs. “This is an even larger issue for special populations of children, including preterm infants and newborns, and in children with chronic or rare diseases.”

According to the AAP, a drug’s off-label status does not imply an improper or experimental use. Health care practitioners caring for children can make therapeutic decisions to use drugs off-label based on expert opinion or on evidence for the medication’s use in a different population.

The passage of the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act has resulted in more than 500 labeling changes, including expanded labeling that includes pediatric information. According to the AAP, the two laws are an “essential first step” in expanding the evidence on use of medications in children, but more work remains to ensure the best possible outcomes for children.

The AAP encourages pediatricians to advocate for research of drugs for children, and supports the publication of drug trials, including negative studies, in academic journals. The AAP also advises that health insurance companies should not use labeling status as the sole criterion to determine whether a medication is eligible to be reimbursed for use in children. Less expensive medications that are considered appropriate for adults should not be automatically considered as first-line treatment in children.